New Drug Demonstrates THE Power of Patient and Research Foundation Advocacy

The F.D.A.'s recent approval of Kalydeco, a drug that targets a mutation carried by approximately 4% of cystic fibrosis patients (about 1,200 people), highlights the importance of patients and research foundations in advocating for cures regardless of how small the receiving patient population may be.

Kalydeco was developed by Vertex Pharmaceuticals with significant support from the Cystic Fibrosis Foundation, which provided $75M over a decade ago to galvanize the search for small molecules that improve the function of the protein mutated in cystic fibrosis.  In a news release by the FDA, Commissioner Margaret A. Hamburg, M.D. said, “The unique and mutually beneficial partnership that led to the approval of Kalydeco serves as a great model for what companies and patient groups can achieve if they collaborate on drug development.”

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